A new study has found that patients with sickle cell disease (SCD) have little knowledge and neutral attitudes about gene therapies and other emerging treatments for their condition.

The study, published in Pediatric Blood & Cancer, found an association between greater knowledge of the treatments and a belief that they are safe.

“Since little is known about patient or parent/caregiver’s knowledge, attitudes, and beliefs toward hematopoietic cell transplantation [HCT] and gene therapies for SCD, we sought to conduct a needs assessment of young adult patients and parental caregivers to compare responses alongside two established treatments for SCD (chronic transfusion therapy and hydroxyurea),” the authors wrote.

The research team conducted a survey at 2 centers in the US on 66 people with SCD aged 13 years or older, most of whom identified as Black, and 38 caregivers. The survey covered attitudes and knowledge of common treatments such as hydroxyurea and transfusion therapy and emerging treatments such as HCT and gene therapies for SCD.

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The results revealed very little knowledge regarding bone marrow transplantation and gene therapy as potentially curative treatments in SCD. Furthermore, only 21% of the patients demonstrated sufficient health literacy as determined by the results of the Newest Vital Sign literacy assessment tool. However, there was no relationship between health literacy level and knowledge of bone marrow transplantation or gene therapies.

The authors did find an association between increased knowledge of emerging therapies and a perception of their safety and effectiveness.

The authors urgently recommend increased patient education in a variety of formats and over multiple visits to improve awareness and increase understanding of the various treatment options available to patients with SCD. They further recommend including patients in the design of such interventions, as well as the chance to meet and interact with other patients who have experienced these treatments with the aim of enhancing their acceptability.


Sharma A, Young A, Carroll Y, et al. Gene therapy in sickle cell disease: attitudes and informational needs of patients and caregivers. Ped Blood Cancer. Published online March 5, 2023. doi:10.1002/pbc.30319